Search Results - "Paul Simons, J"
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Systemic Exosomal Delivery of shRNA Minicircles Prevents Parkinsonian Pathology
Published in Molecular therapy (04-12-2019)“…The development of new therapies to slow down or halt the progression of Parkinson’s disease is a health care priority. A key pathological feature is the…”
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Clusterin regulates β-amyloid toxicity via Dickkopf-1-driven induction of the wnt–PCP–JNK pathway
Published in Molecular psychiatry (01-01-2014)“…Although the mechanism of Aβ action in the pathogenesis of Alzheimer’s disease (AD) has remained elusive, it is known to increase the expression of the…”
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Plasmin activity promotes amyloid deposition in a transgenic model of human transthyretin amyloidosis
Published in Nature communications (07-12-2021)“…Cardiac ATTR amyloidosis, a serious but much under-diagnosed form of cardiomyopathy, is caused by deposition of amyloid fibrils derived from the plasma protein…”
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Pathogenetic mechanisms of amyloid A amyloidosis
Published in Proceedings of the National Academy of Sciences - PNAS (01-10-2013)“…Systemic amyloid A (AA) amyloidosis is a serious complication of chronic inflammation. Serum AA protein (SAA), an acute phase plasma protein, is deposited…”
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C‐reactive protein is essential for innate resistance to pneumococcal infection
Published in Immunology (01-07-2014)“…Summary No deficiency of human C‐reactive protein (CRP), or even structural polymorphism of the protein, has yet been reported so its physiological role is not…”
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Systemic exosomal siRNA delivery reduced alpha-synuclein aggregates in brains of transgenic mice
Published in Movement disorders (01-10-2014)Get full text
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Type II PI4-kinases control Weibel-Palade body biogenesis and von Willebrand factor structure in human endothelial cells
Published in Journal of cell science (15-05-2016)“…Weibel-Palade bodies (WPBs) are endothelial storage organelles that mediate the release of molecules involved in thrombosis, inflammation and angiogenesis,…”
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A specific nanobody prevents amyloidogenesis of D76N β2-microglobulin in vitro and modifies its tissue distribution in vivo
Published in Scientific reports (21-04-2017)“…Systemic amyloidosis is caused by misfolding and aggregation of globular proteins in vivo for which effective treatments are urgently needed. Inhibition of…”
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Overexpression of 5-HT2C receptors in forebrain leads to elevated anxiety and hypoactivity
Published in The European journal of neuroscience (01-07-2009)“…The 5‐HT2C receptor has been implicated in mood and eating disorders. In general, it is accepted that 5‐HT2C receptor agonists increase anxiety behaviours and…”
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Class I Major Histocompatibility Complex, the Trojan Horse for Secretion of Amyloidogenic β2-Microglobulin
Published in The Journal of biological chemistry (07-02-2014)“…To form extracellular aggregates, amyloidogenic proteins bypass the intracellular quality control, which normally targets unfolded/aggregated polypeptides…”
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Pharmacological removal of serum amyloid P component from intracerebral plaques and cerebrovascular Aβ amyloid deposits in vivo
Published in Open biology (01-02-2016)“…Human amyloid deposits always contain the normal plasma protein serum amyloid P component (SAP), owing to its avid but reversible binding to all amyloid…”
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Oligonucleotide-mediated gene editing is underestimated in cells expressing mutated green fluorescent protein and is positively associated with target protein expression
Published in The journal of gene medicine (01-02-2012)“…Background Single‐stranded DNA oligonucleotides (ssODNs) can introduce small, specific sequence alterations into genomes. Potential applications include…”
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Systemic exosomal siRNA delivery reduced alpha‐synuclein aggregates in brains of transgenic mice
Published in Movement disorders (01-10-2014)“…ABSTRACT Alpha‐synuclein (α‐Syn) aggregates are the main component of Lewy bodies, which are the characteristic pathological feature in Parkinson's disease…”
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Antibody-Associated Reversal of ATTR Amyloidosis–Related Cardiomyopathy
Published in The New England journal of medicine (08-06-2023)“…The investigators describe antibodies that bound amyloid in three patients with transthyretin amyloidosis. Spontaneous regression of cardiomyopathy occurred in…”
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Targeted In Situ Gene Correction of Dysfunctional APOE Alleles to Produce Atheroprotective Plasma ApoE3 Protein
Published in Cardiology research and practice (01-01-2012)“…Cardiovascular disease is the leading worldwide cause of death. Apolipoprotein E (ApoE) is a 34-kDa circulating glycoprotein, secreted by the liver and…”
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Reducing β-Amyloid by Inhibition of BACE1 : How Low Should You Go?
Published in Biological psychiatry (1969) (15-04-2015)Get full text
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Novel defect in phosphatidylinositol 4‐kinase type 2‐alpha (PI4K2A) at the membrane‐enzyme interface is associated with metabolic cutis laxa
Published in Journal of inherited metabolic disease (01-11-2020)“…Inherited cutis laxa, or inelastic, sagging skin is a genetic condition of premature and generalised connective tissue ageing, affecting various elastic…”
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Exon Skipping of Hepatic APOB Pre-mRNA With Splice-switching Oligonucleotides Reduces LDL Cholesterol In Vivo
Published in Molecular therapy (01-03-2013)“…Familial hypercholesterolemia (FH) is a genetic disorder characterized by extremely high levels of plasma low-density lipoprotein (LDL), due to defective LDL…”
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The effects of short-term JNK inhibition on the survival and growth of aged sympathetic neurons
Published in Neurobiology of aging (01-10-2016)“…Abstract During the course of normal aging, certain populations of nerve growth factor (NGF)-responsive neurons become selectively vulnerable to cell death…”
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Oligonucleotide-directed gene-editing technology: mechanisms and future prospects
Published in Expert opinion on biological therapy (01-03-2012)“…Gene editing, as defined here, uses short synthetic oligonucleotides to introduce small, site-specific changes into mammalian genomes, including repair of…”
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